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gad65 aav vector is a recombinant adeno-associated virus (AAV) vector engineered to express the human glutamate decarboxylase 65 (GAD65) enzyme in target tissues, particularly neurons of the dorsal root ganglion (DRG). Its primary mechanism of action is mediated gene therapy: the vector delivers the GAD65 cDNA, resulting in sustained GAD65 protein expression and the subsequent increase of local gamma-aminobutyric acid (GABA) synthesis. This elevation in GABAergic transmission provides analgesic effects and neuroprotection by counteracting nociceptive neuron hyperactivity, neuroinflammation, and neurodegeneration. The gad65 aav vector is being developed chiefly as a gene therapy for neuropathic pain and is supported by multiple preclinical studies that demonstrate efficacy in animal models of chronic and diabetic neuropathic pain through long-term pain reduction and improvement of neuroinflammatory markers[1][2].
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